Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial-Reference-Cited by-同舟云学术

Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial

Published:2016-01-07 Issue:1 Volume:127 Page:17-28
ISSN:0006-4971
Container-title:Blood
language:en
Short-container-title:

Author:

Bride Karen L.¹²,Vincent Tiffaney¹²,Smith-Whitley Kim²³,Lambert Michele P.²³,Bleesing Jack J.⁴,Seif Alix E.¹²,Manno Catherine S.⁵,Casper James⁶,Grupp Stephan A.¹²,Teachey David T.¹²

Affiliation:

1. Division of Oncology, The Children's Hospital of Philadelphia, Philadelphia, PA;

2. Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA;

3. Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA;

4. Division of Bone Marrow Transplantation and Immune Deficiency, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, University of Cincinnati, Cincinnati, OH;

5. Department of Pediatrics, New York University Langone Medical Center, New York, NY; and

6. Division of Pediatric Hematology, Oncology and Bone Marrow Transplant, Medical College of Wisconsin, Children’s Hospital of Wisconsin, Milwaukee, WI

Abstract

Key Points Sirolimus monotherapy is a safe and effective steroid-sparing agent, improving autoimmune cytopenias in highly refractory patients. Sirolimus is particularly active in ALPS and should be an early therapy option for patients who require chronic therapy.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

Link

http://ashpublications.org/blood/article-pdf/127/1/17/1391837/17.pdf

Reference17 articles.

1. Diagnosis and management of autoimmune cytopenias in childhood.;Teachey;Pediatr Clin North Am,2013

2. Autoimmune and other cytopenias in primary immunodeficiencies: pathomechanisms, novel differential diagnoses, and treatment.;Seidel;Blood,2014

3. Autoimmune lymphoproliferative syndrome: new approaches to diagnosis and management.;Teachey;Clin Adv Hematol Oncol,2011

4. New advances in the diagnosis and treatment of autoimmune lymphoproliferative syndrome.;Teachey;Curr Opin Pediatr,2012

5. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome.;Teachey;Br J Haematol,2009

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